Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca' Granda Maggiore Policlinico Hospital Foundation, Milan, Italy.
Haemophilia. 2018 Jul;24(4):557-562. doi: 10.1111/hae.13519. Epub 2018 May 29.
In the second decade of the third millennium there have been dramatic developments pertaining to the availability of highly innovative drugs for hemophilia care, notwithstanding a satisfactory previous scenario.
I am going to emphasize the role of 2 main categories of players: scientist physicians who produced important translational research and the pharmaceutical industry, who developed, produced and made commercially available so many improved treatment weapons stemming from the translational research of the forementioned scientist physicians.
Pertaining to the role of scientist physicians, I chose to mention first those who were successful in the 1980 in the production of recombinant coagulation factors. In addition, those who more recently helped to produce new non substitutive therapies given by the subcutaneous route, and recombination coagulation factors with an extended half-life.
Current miraculous progress in hemophilia therapy is stemming from the research work of outstanding scientist physicians who acted in close collaboration with small biotechnology companies, leading to the early development of innovative therapeutic products, subsequently taken to the market place by the so called Big Pharma. I shall briefly provide my views to explain the fact that large pharmaceutical companies show more and more interest in such a rare disease as the hemophilias.
在第三个千年的第二个十年，尽管之前的情况令人满意，但在血友病治疗方面，具有创新性的药物的供应有了显著的发展。
我将强调两类主要参与者的作用：一是生产重要转化研究的科学家医生，二是开发、生产和商业化如此多改进治疗方法的制药行业，这些方法源自上述科学家医生的转化研究。
就科学家医生的作用而言，我首先选择提到那些在 20 世纪 80 年代成功生产重组凝血因子的人。此外，还有那些最近帮助开发通过皮下途径给予的新型非替代疗法，以及半衰期延长的重组凝血因子的人。
目前血友病治疗的惊人进展源自杰出科学家医生的研究工作，他们与小型生物技术公司密切合作，早期开发创新治疗产品，随后由所谓的大型制药公司推向市场。我将简要提供我的观点，以解释大型制药公司对血友病等罕见疾病越来越感兴趣的事实。
